On March 9, Acceleron Pharma, Inc., announced that a phase 2 clinical trial of its therapy under development, ACE-083, for the treatment of Charcot-Marie-Tooth disease (CMT) did not show statistically significant improvements in functional tests compared to placebo. As a result, Acceleron is discontinuing development of ACE-083 and its clinical trials program for CMT. Delivered by . . .
At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with Charcot-Marie-Tooth disease (CMT), as well as caregivers and loved ones, to attend the MDA Engage CMT Symposium taking place Nov. 9 in . . .
Even though the Martinez family lives in Burbank, Calif., within an hour’s drive to Los Angeles’ Cedars-Sinai Medical Center, their road to the hospital’s specialized clinics — to Dr. Robert Baloh and his work there — was a long one. Daniel and Gladis Martinez have two daughters: Genesis, 15, and Isabella, 14, born just 10 . . .
Michael Shy, PhD, professor of Neurology and Pediatrics at the University of Iowa, was awarded an MDA Clinical Research Network Grant (CRNG) totaling $423,413 over three years to further develop the Inherited Neuropathies Consortium (INC), a network of clinical investigators dedicated to developing the infrastructure necessary to evaluate therapies for patients with inherited peripheral neuropathies, . . .
Receiving a diagnosis of a neuromuscular disease is often not easy — there can be denial, anger, grief, and a frustrating sense that maybe life is, in a way, over. For Wolfgang Pernice, PhD, however, being diagnosed with Charcot Marie Tooth disease (CMT) in his early teens didn’t bother him that much. He says his . . .
In a study conducted in mice, scientists led by an MDA-funded researcher found that increasing the amount of a certain mitochondrial membrane protein, mitofusin-1 (MFN1), lessened symptoms of Charcot-Marie-Tooth disease (CMT) type 2A. The findings suggest that it may be possible to treat CMT2A using gene-replacement therapies that deliver functional copies of missing or mutated . . .
On Mar. 5, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug designation to Acceleron Pharma’s ACE-083, a locally acting muscle agent, for treating Charcot-Marie-Tooth disease (CMT). Delivered by intramuscular injection, ACE-083 is based on the naturally occurring protein follistatin and is designed to enhance the body’s own promoters of muscle growth, specifically in . . .
Researchers at Neurogene are seeking participants living with Charcot-Marie-Tooth disease (CMT) type 4J to participate in a natural history study. This study aims to better understand disease course so researchers will be able to determine clinically meaningful outcome measures for use in future clinical trials. This five-year study will consist of 10 visits (one visit every six months). . . .
On Mar. 1, the Charcot-Marie-Tooth Association (CMTA) and the Muscular Dystrophy Association (MDA) announced that they have jointly awarded a research grant totaling $276,430 over three years to Kleopas Kleopa, M.D., professor and senior consulting neurologist at the Cyprus Institute of Neurology and Genetics, Cyprus School of Molecular Medicine, in Nicosia, Cyprus. Dr. Kleopa is . . .
Young adults living with Charcot-Marie-Tooth disease: You can help in a new study. Allison Eveler, a senior occupational therapy major at Elizabethtown College in Pennsylvania, is conducting an undergraduate research study to determine the impacts of CMT on the social participation of young adults. Allison, who herself lives with CMT, recognized a need for research . . .